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Last Updated on April 18, 2023 by Shabnam Sengupta
Francisco Mojica, a scientist at the University of Alicante in Spain, discovered CRISPRs first in archaea and later in bacteria. Currently, CRISPR/Cas9 is the most widely used and advanced tool for genome editing among the various CRISPR/Cas systems. CRISPR-Cas9 is a type of gene editing technology. The acronym stands for clustered regularly interspaced short palindromic repeats and CRISPR-associated protein 9. This article will explore the potential use of CRISPR in treating down syndrome. We will also examine the latest research on this topic.
Can CRISPR be helpful in treating down syndrome?
It may. There is a possibility that CRISPR, a type of gene editing technology, can be a method of treatment for down syndrome. Genome editing involves modifying an organism’s DNA, allowing specific genetic material to be added, removed, or altered at targeted locations in the genome. As down syndrome is a result of a chromosomal abnormality, scientists are investigating the potential of CRISPR for correcting this genetic inconsistency and potentially offering a viable treatment option.
How does research support this?
Studies suggest that CRISPR/Cas9 systems can remove a particular segment from the short arm of chromosome 21 (Chr21). It can then replace this segment with a customized DNA structure that includes essential genes for chromatin remodeling. The ultimate goal of this approach is to deactivate the extra copy of Chr21 that is responsible for down syndrome. This process has the potential to deactivate the extra Chr21 present in down syndrome. Additionally, other research has explored various gene editing approaches, including CRISPR, for eliminating the extra chromosome in down syndrome.
Similarly, designing two single guide RNAs (sgRNAs) to specifically target repetitive sequences located in the long arm of chromosome 21 proved effective in inducing multiple DNA cleavages and eliminating the entire chromosome. However, complete chromosome deletion presents a significant challenge due to the difficulty of efficiently inducing multiple DNA cleavages. While stem cells derived from individuals with Down syndrome responded positively to the chromosome-removing strategy in the initial trial, replicating the same outcome in embryos was unsuccessful, most likely due to embryonic cell death resulting from chromosomal deletion.
Recently, researchers proposed two alternative strategies. The proposed recommendations were to deactivate, rather than delete, the additional chromosome 21. Future efforts shall concentrate on determining the optimal quantity of materials, specifically the Cas9 protein, and utilizing extensive evaluation techniques such as whole-genome sequencing (WGS) and other assays to conduct comprehensive assessments and functional evaluations of the entire genome following the treatment.
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